AVTX-801 D-galactose Supplementation in SLC35A2-CDG

Starting soon · Phase 2 · Has a placebo group

Conditions studied: SLC35A2-CDG - Solute Carrier Family 35 Member A2 Congenital Disorder of Glycosylation

In brief

This is a multicenter, randomized, double-blind, placebo-controlled, cross-over study to evaluate the efficacy and safety of AVTX-801 in subjects with SLC35A2-CDG

Key facts

Study ID

NCT05402384

Run by

Eva Morava-Kozicz

People needed

10

Starts

2027-01-01

Expected to finish

2028-03-01

Last updated by the study team

2026-06-29

Who can join

Age: 0 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Molecular confirmation of SLC35A2 genetic variant
• Age > 1 month
• Presence of seizures, chronic vomiting, chronic constipation, or chronic diarrhea
• A parent or legal guardian must be available and willing to provide consent on behalf of minor subjects or adult subjects who are unable to give informed consent due to developmental disabilities.
• Use of contraception in females > age 8 years
• Previously performed eye exam within last year

You may not qualify if…

• Aldolase-B deficiency
• Galactosemia
• Hemolytic uremic syndrome
• Hemoglobin < 7 mg/dL
• LFTs > 3x ULN
• Previously experienced severe AEs from oral galactose (severe diarrhea, vomiting, constipation, galactosuria, or increased liver glycogen storage)
• Other history of galactose intolerance as determined by the investigator
• Currently treated with ketogenic diet
• Current enrollment in another trial involving investigational compounds
• Ongoing dietary D-galactose supplementation
• Use of investigational compounds
• Pregnancy

Where it is running

• Mayo Clinic Minnesota — Rochester, Minnesota, United States

Full record on ClinicalTrials.gov

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